Opportunities for Patient Benefit Using Approved Therapies: The Critical, First-in-Human Trial

Until recently, the process of bringing repurposed drugs to broadly reach patients with unsolved medical needs was well-known to healthcare stakeholders, but not often to the public. The COVID-19 pandemic brought repurposing into the public eye more than ever. But the ease of patients receiving repurposed drugs differs by country. In the US, doctors can prescribe any FDA-approved drug for their patients, regardless of whether that drug has been approved to treat the patient’s condition, called off-label use. Approximately 20% of all prescriptions written in the US are written ‘off-label”. Randomized controlled trials can provide data necessary for physicians (and their patients) to consider off-label drug use as well as for regulatory agencies to approve a drug for a new indication, when appropriate. This session will discuss the critical Phase I or IIA, first-in-human, proof-of-concept or pivotal clinical trial that, when successful, can catalyze the funding required for a follow-on Phase IIB or III trial required for broad off-label use and/or for regulatory approvals.